Finding Answers to the Unimaginable, Together

"There are moments that the words don't reach. There is suffering too terrible to name. You hold your child
as tight as you can, and push away the unimaginable. There are moments you are in too deep, it feels easier to just
swim down....and learn to live with the unimaginable." --It's Quiet Uptown, Hamilton

Sticking with the Hamilton theme from my last post, I've been reflecting on what
it’s like to be the caregiver for someone with a rare, progressive and fatal disease that doesn't have a cure. In
other words, "the unimaginable." We give this disease our all, day in and day out. We sit for hours every day doing
treatments to stay well and yet we're not well. It's not going to stop us from doing them, but it’s a small bit
heartbreaking that his time and efforts feel fruitless. If he's going to get sick anyway, if this disease will
progress in spite of our best efforts, why can't he just play?

We have all of these things to consider, a myriad of decisions to make again, always. What’s going wrong? What can
we test? How will we identify success? I recently read an article in BioMed Central Journal entitled,
“Six ‘biases’ against patients and careers in evidence-based medicine.” To quote the article:

“At the heart of evidence based medicine (EBM) is the patient, whose informed choices have long been recognised as
paramount. However, good evidence-based care is more than choices.”

It goes on to suggest a counter argument to the one that supports a failure to follow EBM carrying a high risk of
danger to patients, stating instead the risks associated with only following evidenced based medical guidelines and
instruction and the opportunities for improving patient outcomes by expanding this view:

“. . . we should embrace patient involvement in research, make more systematic use of individual (‘personally
significant’) evidence, take a more interdisciplinary and humanistic view of consultations, address unequal power
dynamics in health care encounters, support patient communities, and address the inverse care law.”

Having been a caregiver for just over 6 years for my son with cystic fibrosis, I find myself full of expertise that
is born from experience, and yet often without a place to share what I know in any formal capacity to shape his
care or the care of others. Online patients communities like Smart Patients and even Facebook and Twitter offer
unparalleled connection to others like you, sharing real experiences and often data for how they’ve found
improvement in whatever outcomes are most important to them. Sadly, the benefit that I can find from this
information is dependent upon my stumbling upon it at the right moment. Instead:

How might we increase these serendipitous moments that truly do impact health and care?

How can we organize and share what we know in a way that will enable us to find personalized treatments and
faster cures?

What will it take for us to get the right thing to the right person at the right time, every time?

I think that something medicine fails to recognize—perhaps even more specifically something clinical research fails
to recognize—is the difference between the space where endpoints are identified, “a world of controlled
experiments, abstracted variables, objective measurement of pre-defined outcomes, average results, or generalizable
truths,” and the messy, idiosyncratic, and unpredictable world of a particular person in a particular family
context. The risk that individuals are willing to assume, be it for personal, social, cultural or other reasons,
often is not addressed at the time that these decisions are being made. A patient’s ability to participate in a
study is judged against goals that are set for them.

When a study fails to recruit, or when adherence to the study plan falls through, or when there is an adverse event
that isn’t captured, we have traditionally failed to reflect on the process of the flawed trial design, instead
pointing to the patient who has been kept in the dark. ‌We need a clinical research
system that enables learning rather than judgment‌: the patient didn’t fail the
trial, the trial failed the patient.Can we design a trial that fits into those messy, idiosyncratic and
unpredictable lives? Perhaps this is more simple for adult patients who are making decisions independent of some of
the constraints that I feel as a mother caring for my son. I define and redefine the benefit-risk ratio every time
we have to make a decision:

What are the time constraints of the study?

Can we schedule it around his favorite activities?

Are there side effects that will impact him and others in our family?

Does the benefit of participation outweigh the risk?

I know that these thoughts aren’t uniquely mine. During this National Family Caregiver Month, I recently stumbled
upon Caregiving.com’s Six-Word Stories, where
caregivers reflect on different topics related to our caregiving experiences. Stories like “Holding it together for
everyone”, “Listen to me, I know stuff” and “Get me off this hamster wheel” help me to feel connected, to know that
I’m not alone.

I’m honored to be able to share my story with the world, one that challenges the paradigm for how you can work with
patients and caregivers. Our lived experiences with research can help to grow our collective understanding of the
benefits and challenges of participation – what is it that each of us want and how can we collaborate to
achieve that.

As Desmond Tutu said, “There comes a point where we need to stop just pulling people out of the river. We need to
go upstream and find out why they’re falling in.” While the contributions of health care providers and research
have historically been focused on “pulling people out of the river,” the patient advocates are inviting the rest of
the health care community to take our hand and walk with us upstream, to learn from us and with us. Together we can
make the shift from reactive to proactive care. Together we find answers and change outcomes.